罕見病發(fā)病率低、病情復雜、診斷難度大，導致其治療藥物研發(fā)面臨諸多困難。為滿足臨床迫切需求，鼓勵罕見病藥物研發(fā)，近年來國家出臺一系列政策及技術指導原則。梳理了2015年至今的罕見病治療藥物研發(fā)激勵政策，通過查閱文獻及公開資料，整理2018—2023年批準上市的用于治療第一批罕見病目錄中罕見病治療的藥物信息，分析中國罕見病藥物研發(fā)現(xiàn)狀。建議定期更新罕見病目錄，加強罕見病藥物研發(fā)者權益保護，鼓勵優(yōu)質罕見病藥物仿制，以推動罕見病藥物研發(fā)產業(yè)創(chuàng)新發(fā)展、滿足罕見病患者的用藥需求。

The low incidence, complexity and diagnosis difficulty of rare disease lead to the difficulties in the research and development of drugs for rare diseases. To meet the urgent clinical demands and encourage the research of drugs for rare diseases in China,Chinese government has issued a series of policies and technical guidance. The policies for the research and development of drugs for rare diseases is combed from 2015. By consulting the relevant literature and public information, drug approvals for rare diseases listed in the first catalogue from 2018 to 2023 are presented, then the research and development states of orphan drug is analyzed and summarized. It is suggested that the catalogue of rare diseases should be updated regularly, the rights and interests of drug developers for rare diseases should be protected, the high-quality imitation of drugs for rare diseases should be encouraged, so as to promote the development of the pharmaceutical industry for rare diseases and meet the drugs needs of patients with rare diseases.

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